2015 Top 10 Retinal-Research Advances

The Foundation Fighting Blindness’ scientists, donors and volunteers made 2015 an outstanding year in our fight against blindness. As I tabulated the year’s top 10 research advances—all made possible through FFB funding—I realized that eight are for clinical trials of emerging therapies that are launching or underway.

Moving a treatment out of the lab and into a clinical trial is very challenging; it requires significant financial resources and drug-development knowledge. But advancing a therapy to this critical stage also brings it much closer to the people with retinal diseases who desperately need it. These trials only happen because of the time, money and expertise invested by the Foundation’s supporters.

You can’t wrap a bow around research progress, but I can’t think of a better holiday gift for those hard-working, well-deserving members of the Foundation’s family. So, without further ado, here’s a summary of the year’s top 10 advances made possible by FFB funding:

1. RetroSense Therapeutics received FDA authorization to launch a clinical trial of its optogenetic gene therapy. By harnessing surviving cells in the retina, the treatment holds promise for restoring some vision to people who are completely blind, regardless of the gene mutation causing their disease.

3. ReNeuron has received FDA authorization to launch a clinical trial of its stem-cell therapy for people with RP. Researchers believe the treatment can save and restore vision by replacing lost photoreceptors and keeping existing ones healthy. This treatment has the potential to treat many retinal degenerative conditions, including Usher syndrome, because it might be used to replace photoreceptors regardless of the gene mutation causing the disease.

4. The University of California, Irvine, in collaboration with the company jCyte, has started a clinical trial of a stem-cell therapy designed to preserve and rescue photoreceptors in people with RP. The treatment might also work for other diseases, including Usher syndrome.

8. The Foundation and the biopharmaceutical company Vision Medicines are partnering to develop a drug known as VM200 for preserving vision in people with Stargardt disease. FFB is committing up to $7.5 million to co-fund VM200’s development. Vision Medicines plans to launch a human study of the therapy in 2016.

9. In a Phase I clinical trial, Oxford BioMedica’s RetinoStat® gene therapy for wet AMD stabilized vision in 21 patients over a 48-week period. As opposed to other wet AMD treatments which require monthly injections, a single injection of RetinoStat may last several years.

In 2015, the Foundation also continued to fund early lab studies for identifying new therapeutic approaches. We currently fund 106 projects at 61 prominent institutions around the world. These efforts include advancement of gene, stem-cell and pharmaceutical therapies as well as oversight of natural history studies and identification of outcome measures, both of which aid in the design and successful execution of clinical trials.

The Foundation’s growing patient registry, My Retina Tracker, is now being used by several companies to identify potential clinical-trial participants.

As we move into the new year, please stay tuned to the Foundation’s website, blog and other publications for the latest news on our research progress.

Most of all, I want to extend to the Foundation’s supporters my sincere gratitude for all they do to drive vision-saving treatments and cures.

57 Responses to '2015 Top 10 Retinal-Research Advances'

My son is 15 years old , he was diagnosed unfortunately with retinitis pegmentosa when he was 8 years old. He is under vitamin A 10000 iu. He is stable during the day however in night time and darkness, as you know the vision is weak and can’t focus. The good thing that since he was diagnosed the is no negative deterioration.
Accordingly, I would appreciate providing me with the updates on this disease and the possibility of treatment .

Dear Mustafa, You should consider genetic testing for your son to identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

Whether your son’s disease gene is identified or not, you should still consider enrolling him in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. I hope you find this information useful and thank you for your support.

HI, ensure the vitamin A is the dry tablet form, palmitate – solgar do one, this may be what you are using.

Other things which help general eye health are Retinex (containing Lutein) and Bilberry. Bilberry is made from the berries and was used by pilots in the war to help their night vision. Ensure tablets are made from “extract” and not just powder.

Hoping that eventually a treatment will be genetic therapy or stem cell.

hello sir how are you just now i read your this and same disease i have same problems and so much problem in night or sharp sunlight i am consult many doctors they all says no treatment available in the world.so please tell me if any possible soulation for me lots of thanks please no mention anything on web page thanks my email id if you send me any information sabakiran360@gmail.com

Good morning and happy Christmas. if not write to its place but I would tell you something about my son. He is 18 and suffering from advanced Retinitis Pigmentosa that makes it difficult to attend to the college. we are available from Argentina to what you see fit. Sorry if no suitable place to send this data. I wish you the best. thanks a lot.

Dear Dr. Masuelli, I am sorry to hear of your son’s diagnosis. He should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted on the gene or protein. With a molecular diagnosis, he may qualify for one of the gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
Whether his disease gene is identified or not, he should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/
It may also be helpful for your son to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. I hope you find this information helpful and thank you for your support.

Retinopathy of prematurity (ROP) is a potentially blinding eye disorder that primarily affects premature infants weighing about 2¾ pounds (1250 grams) or less that are born before 31 weeks of gestation (A full-term pregnancy has a gestation of 38–42 weeks). The smaller a baby is at birth, the more likely that baby is to develop ROP. This disorder—which usually develops in both eyes—is one of the most common causes of visual loss in childhood and can lead to lifelong vision impairment and blindness. It is very likely that some of the treatments being developed to treat RP and AMD may have some utility in treating ROP. Currently, there are several clinical trials being conducted for ROP. Here is a web link: https://clinicaltrials.gov/ct2/results?term=retinopathy+of+prematurity&Search=Search

You should know that there are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:

You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

Below is a list of hospitals in India that have experience in treating patients with retinal disease:

Vitamin A in the dry tablet form (palmitate) – solgar do one, 10000 – 15000 IU a day. This is currently the only recommended “treatment” and in trials a few years ago it was said to help slow the progression of retinitis.
I think I read it also helps with glare from bright lights at night.

Other things which help general eye health are Retinex (containing Lutein) and Bilberry. Bilberry is made from the berries and was used by pilots in the war to help their night vision. Ensure tablets are made from “extract” and not just powder.

Hoping that eventually a treatment will be genetic therapy or stem cell.

Hi,
My daughter is recently diagnosed with RP. She is only 15 yrs old . It was an incidentally finding during her eye check up . She is myopic .
Her problem at this stage is seeing clearly in darkness . She has not complained of any day time or dim light difficulty .
I would really like to know if there is a treatment to stop the progression of this disease at this stage and to restore the lost vision .
I am really hoping on finding a treatment for this devastating disease.
Please help

You should also consider enrolling your daughter in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

Finally, you may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

DEAR SIR,
HOW ARE YOU,
MY 19 YEAR OLD SON IS RECENTLY DIGNOSED BY RP.HE IS FACING DIFFICULTY IN SEEING AT NIGHT AND DURING DIM LIGHT.ALSO HIS DAY VISION IS NARROWING AND HE CAN SEE ONLY FROM THE CENTRE OF HIS EYES ONLY.
HE IS TAKING AQUASOL A ,ONE CAPSULE PER DAY.
PLEASE SUGGEST ME IF THERE IS ANY REMEDY FOR THIS PROBLEM.
I AMD VERY DEPRESSED FOR HIM SINCE I CAME TO KNOW ABOUT HIS DISEAS.
I AM FROM JAIPUR INDIA
R.AGRAWAL

Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

I Don’t know how long it will take to find a proper treatment of this drastic disease called RP.
But I am very hopeful as I can see the doctors and scientists are working with FFB to remove this dreadly disease existence.
I pray God almighty will support all of you people who are working day and night to fight blindness.
I wish Year 2016 will be a success and will set landmark for it.
thanks and regards
R.Agrawal

You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
Whether the disease gene is identified or not, you should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/
It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

Below is a list of institutes in India that have experience in diagnosing and treating RP:

I am a 33y/O woman with RP L. When I was younger, the docs told my mom that I had RP and would be nearly blind by 20. Upon further exams,they discovered I have the rarer, less severe form. Over the past decade my night lines has finally prevented me from driving at night. Are there any trials or medicines available in US? Missouri or the Midwest specifically as have no money for overseas travel to India.

Hi, is there a number I can call to have my son enrolled in some of type trial testing for LCA? My son is 19 years old, he can see up close, small prints or words, the problem is distance -near sided. Thank you for all the work you do. Does eye laser surgery help him?

Hai, FFB:
I hear a lot of discussions on rp in this forum, I want to know is there any on going project related to Staphyloma or Cyliary staphyloma in particular. Your responses will be of great help for my brother and me. Both of us are suffering from total blindness by birth caused by Ciliary Staphyloma.

This is just for information for people suffering from RP . One of my family members has this disease and while there is promise in gene therapy in future we have tried Ayurvedic treatment in Kerala at Sreedharyam (3 weeks course to be repeated around every 6 months) that has helped in stabilizing the vision loss at least. Please see these web sites http://www.sreedhareeyam.com/ and http://www.ayurprakash.com/ are some of facilities offering this treatment . You can consult them over phone and plan accordingly depending upon convenience.

Good day,
Thank you for all of your wonderful work and persistence. My brother was diagnosed with AION in both eyes, with complete blindness in one eye and 5% in the other. Is there any future hope for him?

Anterior ischemic optic neuropathy (AION) is a medical condition involving loss of vision due to damage to the optic nerve from insufficient blood supply. Please see the following link for more information on the disease and possible treatments: http://www.lowvision.org/ischemic_optic_neuropathy.htm

Dear Doctor,
Im
Atul from maharashtra india
My my twins baby when they born very low waight and pretrum,after one month i was check both babys eye all is normal report but after two month both babys eye flow water so im going asia most popoler eye hospital in Hydrabad there Dr said your both babys both eyes retina damaged they do one surgery both babys eye and said this surgery will use when
the retina devlop reserch
So i humble preay to god give sucsess to you
Thanks
Atul Panchal
Nanded,Maharashtra,India.

Dear Paul, You should know that there are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf . You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf . You should also consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/ . Finally, you may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

Hi. I have a friend who was diagnosed with retinitis pigmentosa last 2015. I want to ask if is there any possibility to retain his vision until he gets 50 years old or older than that. He is 22 years old now.

Dear Anazel, It is certainly possible that your friend who is 22, could retain his vision until he is 50 and maybe even until he is ninety. The rate at which retinal disease progresses is determined by a number of factors including: the gene that is mutated, the type of mutation and the presence of modifier genes. Environment, nutrition and health, may also play a role. Your friend should know that there are three inherited forms of RP: recessive, dominant and X-linked. If he is not sure which type he has, he should ask his ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

Your friend should also consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

Finally, your friend may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing provides information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

Best disease, also known as vitelliform macular dystrophy, is an inherited form of macular degeneration characterized by a loss of central vision. For more information on Best disease, please see the following link: http://www.blindness.org/best-disease
You will be happy to know that the Foundation Fighting Blindness is currently funding research efforts at the University of Pennsylvania and University of Wisconsin to develop a gene therapy treatment for Best disease. Already, preliminary data has been published showing that gene therapy can stop disease progression in a dog model. Here is the link: http://www.blindness.org/foundation-news/progress-reported-gene-therapy-development-best-disease. If successful, these research studies will be moved under the jurisdiction of FFB’s Clinical Research Institute which is focused on the development of human clinical trials. There is however, a lot of work to be done before a trial can start. Toxicity and dosing studies in large animals, additional safety studies, generation of a GMP viral vector that can be used in humans and the filing of regulatory paperwork with the FDA are just a few of the things that need to be done before the trial can start. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

I would like to know if there is a Dr out there that could help my father that’s 72 or sister that’s 38 that are almost blind due to a hereditary retinal disease they can’t explain. It also has to do with shape of eye. My grandmother and 4 aunts and uncles have gone completely blind on my dad’s side. It’s scarey. Please help.

Charlene, I am quite confident that your family members can obtain a diagnosis. Below is a web link to a list of retinal specialists in your state. This list was developed to help patients who are outside of the range of The Foundation’s Research Centers find local specialists. To compile this list, The Foundation requested names of all vitreo-retinal specialists from the American Academy of Ophthalmology’s membership. We then wrote to each listed member, asking if they wished to be included on our referral list. Here is the link:http://www.blindness.org/retinal-physicians

There are currently a number of different treatments available for the treatment of Retinopathy of Prematurity or ROP. To read more about ROP and possible treatments, please see the following weblink: https://nei.nih.gov/health/rop

Hi
My name is Motlalepula
When I was 15 years I lost sight IN my left eye after being hospitalised for 2 to three months. I don’t know what caused the problem what I know is that my eye at the time was swollen and bulgrd out of the eye hole. The doctors at that time wanted me to have it removed saying it is going to affect the other eye but my faith told me that one day I will get help.
hoping to hear from you

It is important that you obtain an accurate diagnosis of your eye condition. With this information, you may be able to qualify for one of the many clinical trials that are taking place. If you live in the U.S., please see the following weblink that contains a listing of eye doctors around the country: http://www.blindness.org/retinal-physicians

You should also consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:https://www.myretinatracker.org/

Finally, you may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

my name is Dawit from Ethiopia & I lost my vision fully exept seeing lights around & I want to know if there is any thing I can do to improve or cure my rp problem. Or if there is a place with treatment please inform me.

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